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US Approved Rx

4

US Previously Marketed

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Primary Target

Microsomal triglyceride transfer protein large subunit

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Homozygous familial hypercholesterolemia

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Secondary

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Bristol-Myers Squibb

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Protein

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ChEMBL

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DAILYMED

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MONOCLONAL ANTIBODY

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Other lipid modifying agents

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Search results for "ORPHAN DRUG|Designated/Approved|Treatment of homozygous familial hypercholesterolemia" in comments (approximate match)

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LOMITAPIDE

82KUB0583F

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Status:

US Approved Rx (2012)

First approved in 2012

Class (Stereo):

CHEMICAL (ACHIRAL)

Targets:

Microsomal triglyceride transfer protein large subunit

Conditions:

Homozygous familial hypercholesterolemia

Lomitapide (INN, marketed as Juxtapid in the US and as Lojuxta in the EU) is a drug for the treatment of familial hypercholesterolemia, developed by Aegerion Pharmaceuticals. It has been tested in clinical trials as single treatment and in combinatio...

ns with atorvastatin, ezetimibe and fenofibrate. The US Food and Drug Administration (FDA) approved lomitapide on 21 December 2012, as an orphan drug to reduce LDL cholesterol, total cholesterol, apolipoprotein B, and non-high-density lipoprotein (non-HDL) cholesterol in patients with homozygous familial hypercholesterolemia (HoFH). On 31 May 2013 the European Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion with a unanimous vote recommending a marketing authorization for lomitapide. On 31 July 2013 the European Commission approved lomitapide as an adjunct to a low-fat diet and other lipid-lowering medicinal products with or without low density lipoprotein (LDL) apheresis in adult patients with HoFH. UXTAPID directly binds and inhibits microsomal triglyceride transfer protein (MTP), which resides in the lumen of the endoplasmic reticulum, thereby preventing the assembly of apo B containing lipoproteins in enterocytes and hepatocytes. This inhibits the synthesis of chylomicrons and VLDL. The inhibition of the synthesis of VLDL leads to reduced levels of plasma LDL-C.

EVINACUMAB

T8B2ORP1DW

Subunit 1 (453) Similarity Search Subunit 2 (453) Similarity Search Subunit 3 (214) Similarity Search Subunit 4 (214) Similarity Search

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Subunit 1 (453) Similarity Search Subunit 2 (453) Similarity Search Subunit 3 (214) Similarity Search Subunit 4 (214) Similarity Search

Status:

US Approved Rx (2021)

First approved in 2021

Class:

PROTEIN

EVOLOCUMAB

LKC0U3A8NJ

Subunit 1 (441) Similarity Search Subunit 2 (441) Similarity Search Subunit 3 (215) Similarity Search Subunit 4 (215) Similarity Search

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Subunit 1 (441) Similarity Search Subunit 2 (441) Similarity Search Subunit 3 (215) Similarity Search Subunit 4 (215) Similarity Search

Status:

US Approved Rx (2015)

First approved in 2015

Class:

PROTEIN

ALIROCUMAB

PP0SHH6V16

Subunit 1 (447) Similarity Search Subunit 2 (447) Similarity Search Subunit 3 (220) Similarity Search Subunit 4 (220) Similarity Search

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Sequence Search

Subunit 1 (447) Similarity Search Subunit 2 (447) Similarity Search Subunit 3 (220) Similarity Search Subunit 4 (220) Similarity Search

Status:

US Approved Rx (2015)

First approved in 2015

Class:

PROTEIN

MIPOMERSEN

9GJ8S4GU0M

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Status:

US Previously Marketed

First approved in 2013

Class:

NUCLEIC ACID

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